Latest Drugwonks' Blog
Ice T?
Testosterone gels – do they provide an important treatment option? Is “Low T” a real disease or just a marketing opportunity? Do these two options need to be mutually exclusive? (The F.D.A. has approved testosterone gels “for use in men who either no longer produce the male sex hormone testosterone or produce it in very low amounts.”)
And what does this have to do with asthma inhalers and DTC?
Interesting article in today’s New York Times (see here).
One error that needs immediate correction. After reporting on the amount of money companies such as Eli Lilly and AbbVie spend on advertising their testosterone gel products, the Gray Lady reports that,
In response to an article Sunday in The New York Times on prescription drug costs for asthma medicines in the United States, a number of readers complained about the high price of inhalers, and that the costs were inflated by the millions of dollars pharmaceutical companies spend on advertising for them.
Except that assumption is not true. Whether or not you agree with the style or substance of pharmaceutical direct-to-consumer advertising, one thing that’s just an economic fact is that advertising budgets do not impact the list price of the product.
Study after study after study done by charitable foundations, the federal government and industry all show that the price of a drug does not correlate to the amount spent on advertising.
In other words, if you look at four medicines that treat cholesterol and compare their advertising budgets, the one that spends the most is not necessarily the most expensive -- and often the reverse is true.
Consider this: According to Tufts University it costs about one billion dollars to bring a new medicine to market. That's one billion per drug -- and those are the ones that make it to market.
Now compare that to the $2.5 billion per year the industry spends on advertising and you have a somewhat better perspective. Reducing direct-to-consumer drug advertising would not reduce the price of drugs.
A reduction in advertising wouldn't reduce the price of medicines, but it would most certainly reduce the number of people visiting their doctors, and I cannot imagine that anyone thinks that is a good thing.
Here's some data from an FDA research study: Doctors do not prescribe medicines just because their patients ask for them after seeing an advertisement. They're prescribing medicines because their patients need them. That's called progress.
An excellent article from BioWorld on the continuing saga on biosimilars from Sacramento to Singapore.
Some snippets …
The U.S. Battle of Biosimilars Continues on Multiple Fronts
By Mari Serebrov, Washington Editor
While the first biosimilars to hit the U.S. are still in development, the battle over how and when the follow-on biologics should be marketed continues to rage on both the local and global fronts.
A skirmish in California ended Saturday when Gov. Jerry Brown vetoed a bill, overwhelmingly passed by state lawmakers, that would limit automatic biosimilar substitution to those the FDA deems “interchangeable.” SB 598 also would have required pharmacists to notify physicians when an interchangeable is substituted for a prescribed biologic.
“Obviously, the governor gave himself a little wiggle room here,” Peter Pitts, president of the Center for Medicine in the Public Interest, told BioWorld Today.
Pitts disagreed with the governor about the timing of the bill. “It’s never too early to say we need to educate doctors and patients about what’s going on,” he said, stressing the importance of promoting safety and pharmacovigilance in the use of biosimilars.
“The untrained ear hears ‘identical’” when biosimilars are mentioned, Pitts said, but the follow-ons are not the same as generic drugs. Noting that even generics, which are supposed to be identical to the reference drug, can have variants that could affect an individual patient’s response, he pointed out that due to their complexity, biosimilars could produce more variances.
Global Battle
While the substitution battle continues on the state level, another biosimilar war is raging on the global front as the countries negotiating the Trans-Pacific Partnership (TPP) push to finalize the trade talks this year. Despite efforts to have the agreement reflect the 12 years of data protection that biologic innovators enjoy in the U.S., the issue hasn’t made it to the 3-year-old negotiating table yet.
Opposition to including the data protection in the TPP is framed, once again, as an issue of increased access. But it really comes down to some of the partners wanting to maximize their own opportunities, Pitts said.
He noted that the 100 most essential drugs identified by the World Health Organization are all off patent, which means they’re available as generics. But people in many countries still don’t have access to those essential small-molecule drugs, so Pitts questioned the assumption that reducing or removing biologic data exclusivity would increase global access to biosimilars, which are more complex and costly than generics.
The complete article can be found here.
California Governor Jerry brown has vetoed SB 598, a bill (passed by both houses of the legislature with overwhelming bipartisan support) that would have allowed biosimilars to be substituted by pharmacists if the F.D.A. deemed the biosimilar “interchangeable” with the reference product.
The California bill would allow substitution of a biosimilar for an innovator product only if FDA declared the biosimilar interchangeable for the specific use; the prescriber had not expressly prohibited use of a biosimilar; the substitution was communicated to patients; the cost to the patient was the same or less than the innovator product; and the pharmacist notified the prescribing physician within five days. The requirement for physician notification would sunset after three years Additionally, the California State Board of Pharmacy would maintain a list of biosimilar products FDA determines to be interchangeable on its website
Patient information? Pharmacist empowerment? Who could be against such a significant public health double play?
According to Governor Brown,
“Doctors with whom I have spoken would welcome this information. CALPERS and other large purchasers warn that the requirement itself would cast doubt on the safety and desirability of more cost-effective alternatives to biologics.”
(CALPERS is the California Public Employees’ Retirement System, which provides health benefits to more than 1.3 million people.)
“Case doubt on the safety …”
Perhaps the Governor should be made aware of what bioequivalence really means.
Per a report in the New York Times, “Ralph G. Neas, president of the Generic Pharmaceutical Association, celebrated the veto, saying that Mr. Brown had “demonstrated compassion for millions of patients and strong fiscal stewardship for the state of California.”
Nothing like celebrating the victory of cost-centric medicine over patient-centric care.
For shame.
The Washington Post reports that Senator Joe Manchin (D/WV),
... is calling for an investigation of “pay to play” allegations regarding a scientific panel that shaped the Food and Drug Administration’s thinking on painkillers and that, according to the organizer’s e-mails, was funded by major pharmaceutical companies that put up as much $25,000 to attend a meeting.
FDA responds that the sessions,
… amounted to what one agency officials described as “an essential collaborative effort.”
And, yes -- collaboration is essential. FDA must be both regulator and colleague. But should money have been charged (by a private, for-profit organization) for industry to have a seat at the table? That’s a good question.
It’s also important to clarify that these were not (repeat – not) advisory committee meetings.
Sent: Thursday, October 10, 2013 02:33 PM
To: FDA-CDER-wide
Subject: Retirement Rumors
CDER Staff:
I want to assure you that I am not planning to retire as erroneously reported in the media today. In fact, quite the opposite is true. I am becoming more deeply involved in many of the Center’s issues, including the proposed reorganizations of the Office of Pharmaceutical Quality and the Office of Generic Drugs.
The inaccuracy of the media has unnecessarily raised concerns among Center staff, and even among my own family. My daughter emailed me this morning to ask if I’m retiring! I continue to be fully committed to the important work CDER does and to its staff who work so diligently to protect the health of the American public.
Janet Woodcock
In July 2013, the Oncology Nursing Society (ONS) held its annual Leadership Weekend. Each year, the organization collects information about oncology nurses, their interests, and their needs. This year, they asked about their membership’s experiences helping patients manage adherence to oral therapies and about their interest in non-CNE training on the topic. The results of the survey, completed by 127 nurses, show that:
• Oncology nurses are experiencing patient non-adherence to oral therapies
• Oncology nurses want and need specific clinical practice assistance to help improve adherence
• Oncology nurses want and will attend educational programs on adherence to oral therapies.
Some findings of interest and importance:
Most Nurses Observe Problems with Adherence
There is evidence that most nurses are observing problems with adherence. Forty-one respondents (32%) said that 25%–50% of their patients have trouble, and another 26 (20%) said that 51%–75% of their patients do. In other words, more than half the respondents said that at least 25% of their patients do not adhere properly to their regimens.
Nurses Highlight Most Common Barriers to Adherence
The nurses ranked various potential causes of non-adherence by their importance to their patients. Cost was viewed as very important by 73 (57%) and important by 31 (24%). Thus, of the 127 total respondents, 104 find that cost, including reimbursement issues and high copays, is a significant barrier to adherence to oral therapies.
Another barrier appears to be side-effect management. Fifty-nine percent said this is an important problem (45 [35%]) or very important (31 [24%]). Cognitive difficulties following instructions are another potential barrier, but perhaps less significant. Although forty-eight (38%) ranked cognitive difficulties as 3 on a 1-5 scale, or of modest importance, another 51 (40%) viewed cognitive difficulties as important or very important in interfering with adherence to oral therapies. Difficulty swallowing seemed to be the least significant barrier, with 55 (43%) suggesting that it was not important or of minimal importance. Twenty-eight (22%) said it was mildly important, and 41 said it was important or very important. Thus, although most of the nurses did not view swallowing difficulty as a critical barrier, it does affect a fair number of patients.
What Do Nurses Need to Help Manage Adherence?
The participating nurses were offered a number of options for possible practical tools or specific aid. They are listed below in order of popularity with the respondents.
• Co-pay and reimbursement assistance was the most positively received form of help with adherence to oral therapies. Here, 119 (94%) of the respondents said they would find the help useful or very useful (70%).
• Side-effect management guides/tear sheets were seen as useful or very useful by another 116 nurses (91%).
• Patient journals/calendars were seen to be useful or very useful by 103 (81%) of the respondents.
• Online resources for both nurses and patients were very positively received, with 100 (79%) respondents viewing them as useful or very useful.
• Patient refrigerator magnets with significant information were also popular, with 72 (57%) indicating they would be useful or very useful.
• Telephone or email follow-up services by drug company or pharmacy were viewed as useful or very useful by 73 nurses (58%).
• Tablet or smartphone apps might be an emerging area of opportunity for drug companies or pharmacies. Fifty-seven (45%) thought they would be useful or very useful.
It’s interesting that the current outreach efforts by pharmaceutical companies and pharmacies rank only one percentage point higher than refrigerator magnets. They’re both 20th century technologies.
What’s also important is that the research differentiated between “online resources” and apps. While both are 21st century technologies, “online” is the substrate -- apps are the wave of the present.
Apps remind, cajole, educate, praise, incentivize, and assist patients in their quest for better health. Apps are at the nexus of safe use, treatment outcomes, and patient satisfaction. And it’s not science fiction.
At present, there are some 17,828 healthcare and fitness apps and 14,558 that can be deemed “medical.” While some are better than others, these numbers tell us one thing – this is not a fad or a trend. It is reality.
I have been working with the Mobile Health Library to help develop their safe use and outcomes support platform. It’s delivered as an app (viable on all tablet and smart phone systems) specifically to address many of the issues called out specifically by the ONS study – facilitating co-pay cards, patient diary functionality, side-effect management, direct physician office communications (via phone, e-mail, and text) and, perhaps most importantly, providing educational services via app-enabled printed materials, website links, and video).
Will our socio-economic “technology gap” lead to a more pronounced “adherence/compliance gap?” It’s an important question. That’s why it’s crucial we remember there is no one-size-fits all solution. But that mustn’t mean we disregard the reality of the growth and pervasiveness of apps, mobile apps. Let’s face it, when it comes to mobile phones, any gap is rather narrow – and it’s getting narrower all the time, literally every day.
The ONS research refers to apps as an “emerging opportunity.” And that’s true. But maybe a better way to phrase it is as an “emergent opportunity.”
Apps are here now. People are using them – more every day, and that includes healthcare providers, patients, and care-givers.
And as Philip K. Dick wrote, “Reality is that which, when you stop believing in it, doesn’t go away.”
The only thing that dies harder than a bad idea is a bad idea with political resonance.
The Wall Street Journal reports:
AUGUSTA, Maine—The hunt for cheaper prescription drugs long has led consumers to reach beyond U.S. borders, but under a Maine law set to take effect Wednesday, their search now will have the state's blessing.
The law, the first of its kind, sanctions the direct purchase of mail-order drugs from some foreign pharmacies. It has ignited a court battle with the pharmaceutical industry and set the stage for a broader fight over access to less-costly medication.
Vendor of choice – CanaRx.
This is particularly appalling since the drugs being sent to U.S. customers from CanaRX are most certainly not “the same drugs Canadians get.” That bit of rhetoric is just plain wrong. CanaRX – by their own admission – sources their drugs from the European Union. And while they may say their drugs come from the United Kingdom, let’s not conveniently forget that 20% of all the medicines sold in the UK are parallel imported from other nations in the EU – like Spain, Greece, Portugal, and Lithuania.
PS/ The drugs CanaRX sells to Americans aren’t even legal for sale in Canada.
Once again -- a brief primer on why drug importation is a bad idea
(1) It doesn’t save money.
(2) The drugs being sent to U.S. customers from Canadian Internet pharmacies are not “the same drugs Canadians get.”
(3) The state experience has been dismal and politically embarrassing.
(4) National Security concerns.
Let’s look at each of these four items.
(1) It won’t save any money. Let’s not forget the non-partisan CBO study that showed that such policy would reduce our nation’s spending on prescription medicines a whopping 0.1% -- and that’s not including the millions of dollars the FDA would need to set up a monitoring system.
(2) The drugs being sent to U.S. customers from Canadian internet pharmacies are not “the same drugs Canadians get.” That bit of rhetoric is just plain wrong. In fact, drugs sold to Americans by Canadian Internet pharmacies aren’t even legal for sale in Canada. This isn’t about the quality of Canadian drug regulation. Canadian Internet pharmacies – by their own admission – are sourcing the drugs they're sending to the United States from outside of Canada. And while they may say their drugs come from Great Britain, let’s not conveniently forget that 20% of all the medicines sold in the UK are parallel imported from other nations in the EU – like Spain, Greece, Portugal, and Lithuania.
And the important political point here is that when Americans are asked if they want drugs from nations other than Canada – the answer is a resounding “no thank you.”
(3) The state experience has been dismal and politically embarrassing. Remember Illinois’ high profile “I-Save-RX”program? Over 19 months of operation, a grand total of 3,689 Illinois residents used the program -- which equals approximately .02% of the population.
And what of Minnesota’s RxConnect program? According to its latest statistics, Minnesota RxConnect fills about 138 prescriptions a month. That's for the whole state. Minnesota population: 5,167,101.
That's not a surprise considering that Minnesota, state officials observed Canadian Internet pharmacies engaging in dangerous practices.
One pharmacy had its pharmacists check 100 new prescriptions or 300 refill prescriptions per hour, a volume so high that there is no way to assure safety.
One pharmacy failed to label its products and several others failed to send any patient drug information to patients receiving prescription drugs.
Drugs requiring refrigeration were being shipped un-refrigerated with no evidence that the products would remain stable.
One pharmacy had no policy in place for drug recalls. Representatives of the pharmacy allegedly said that the patient could contact the pharmacy about a recall "if they wished."
The FDA launched an investigation, confiscating thousands of drug shipments headed for the United States. Some of them were headed for Minnesotans who ordered them over the state's Web site.
When opened, nearly half claimed to be of Canadian origin, but "85 percent of them were from 27 other countries including Iran, Ecuador and China." And 30 of them were counterfeit.
One Minnesota resident discovered that one of his "Canadian" drugs came from Greece, and another came from Vanuatu, a small island in the South Pacific. "I never heard of the place," he said.
Wisconsin also has an importation program, modeled on the one in Minnesota. It too hawks its promise and hides its dangers. All of the legalese buries the fact that the state doesn't accept any responsibility for the safety or effectiveness of any medicines bought on the state's Web site.
The state won't even guarantee that the drugs ordered are what the customer will receive. Not only that, but the state also says that it will not accept any legal responsibility or liability should any of the drugs cause a problem.
And remember Springfield, MA and “the New Boston Tea Party?” Well the city of Springfield is now out of the drugs from Canada business.
(4) National Security concerns. According to a report from the federal Joint Terrorism Task Force, a global terrorist ring with ties to Hezbollah, is importing counterfeit drugs into America by way of Canada. They are doing so for profit today - but could just as easily do so for more nefarious and deadly purposes. And legalizing importation would only facilitate such actions.
And then there are those politically pesky safety issues.
Adding fuel to the reality is a new by the European Alliance for Access to Safe Medicines. The title says it all, “The Counterfeiting Superhighway.”
The report reveals the scope of the unregulated trade of fake pharmaceuticals. Through extensive research and examination of over 100 online pharmacies and over 30 commonly purchased prescription-only medicines, the report makes one thing very clear – we’re not winning the battle.
Key findings from this report
* 62% of medicines purchased online are fake or substandard (including medicines indicated to treat serious conditions such as cardiovascular and respiratory disease, neurological disorders, and mental health conditions).
* 95.6% of online pharmacies researched are operating illegally.
* 94% of websites do not have a named, verifiable pharmacist.
* Over 90% of websites supply prescription-only medicines without a prescription.
* 78.8 of websites violate intellectual property.
My favorite anecdote is the report’s example of an Internet pharmacy whose products came wrapped in pages from the Mumbai Daily News. The most frightening fact, though, is most of the fake medicines “were delivered in seemingly authentic boxes, accompanied by patient information leaflets in good condition and ostensibly trustworthy blister packs.”
"Those who cannot learn from history are doomed to repeat it."
Another way the Republic of South Africa is trying to reinvent itself is in the way it regulates and reimburses for medicines. “BE” means not only “Black Empowerment” (in the post-apartheid sense) but also “bioequivalence.”
I’ve just returned from the “New Developments in Drug Regulation” conference in Pretoria, and there was a lot of serious discussion as to how government regulators can do a better job in a uniquely South African situation.
Some memorable moments …
Tomas Salmonson (Chair of the EMA’s CHMP) told the audience that, while he is a strong believer in transparency, he is not in favor of patient representatives on decision panels. Such representation, he said, “would be like having an elected parliament and then additional members.” He also does not believe that these meetings (akin in many ways to an FDA advisory committee meeting) should be open to the public. I pointed out that having patient reps and open adcomm meetings was a crucial part of the FDA process. Salmonson commented, “I know.”
Transparency translates in different ways. Salmonson noted that one way the EMA promulgates transparency is through its EPARS (European Public Assessment Reports System). The European Medicines Agency publishes an EPAR for every medicine granted a central marketing authorization by the European Commission. EPARs are full scientific assessment reports of medicines authorized at a European Union level.
Salmonson also made the point that a transparent process behind benefit/risk allows consumers and healthcare providers to trust the regulator. FDA – attention must be paid.
Peter Bachmann, head of the Germany’s BfArM (Federal Institute for Drugs and Medical Devices) Coordination Group Unit, spoke to the EMA’s philosophy of being “united in diversity” (a theme that certainly resonated in Pretoria). He also discussed the EMA’s program of mutual recognition, detailing the agency’s methodology of “work-sharing” – a process of cross-national regulatory peer review that should be studied by FDA policy panjandrums for many reasons, not the least of which is quality control.
Ngokoana Khomo, Vice Chair of South Africa’s MCC (Medicines Control Council – the South African version of the FDA) honestly spoke of the tension between “the policy and the practice” of medicines regulation in South Africa. Unspoken was the obvious under-current of a third “p” – politics. And perhaps a fourth – “Potemkin Regulation.” She spoke of the MCC’s mission as “access, equity, efficiency, quality, and sustainability.” Dr. Khomo wisely said that “transparency takes away all suspicions.” But saying and doing is not always the same thing. She also spoke about the rational use of medicine. After all, you can’t spell Ngokoana without NGO.
Ekkehard Baader (Senior Director, Head of EU Regulatory Affairs at Teva) addressed the importance of (and distinctions between) consultation, communications, and cooperation.
Speaking of Teva – there was no discussion of generic drugs during the conference. Not a single mention of API sourcing and quality, not a word about excipients. Disturbing considering the venue and the reality.
BfArM’s Birka Lehmann (a member of PDCO, the EMA’s Paediatric Committee) spoke about issues surrounding informed consent for children (“What if the baby is crying?”) as well as the need for a Paediatric Investigation Plan (PIP) at end of phase 1. Clearly an area ripe for FDA harmonization conversations. Clearly the pediatric train is moving – but EMA and FDA should be on the same track.
At this point you may be wondering what any of this has to do with regulatory reform in the Republic of South Africa. That question was also on the minds of many in the audience who asked questions like, “how much does all of this cost?” and “how many people do you need?” and “how do you train staff?”
Day 2 began with a focus on biosimilars and the successes and failures of the EMA experience – as well as a nod to the work being done by the WHO.
The presentation by Chris Holloway (Group Director of Regulatory Affairs & Chief Scientific Officer at the ERA Consulting Group) was the first person to use the word “quality” – and “process.” He spoke about “balancing needs against expectations.” And it was a welcome addition to the conversation. A breath of real-world reality vs. regulatory theory.
Elwyn Griffiths (WHO) likened his organization’s attempts to develop criteria for biosimilars to the League of Nations – but in a good way.
Haile Selassie, call your office.
Max Wegner (VP, Head of Global Regulatory Affairs, General Medicine at Bayer AG) addressed the unintended consequences of the EMA’s PRAC (Pharmacovigilance Risk Assessment Committee), specifically the impact of public reports of adverse events that had yet to be investigated. I added that the FDA had similar issues with Early Safety Signal Communications and the resultant unintended (but not entirely unsurprising) over reaction by the media and the ensuing leap in non-adherence.
Finally, Marc Blockman of the host Medicines Control Council (MCC) spoke on the issues surrounding both the importance of and difficulties with pharmacoviginance in South Africa. Two of his comments stand out: “Pharmacovigilance is a public service” Bravo. And, “Yes, we have a yellow card – but the form is as much the problem as it is the process.” Sounds familiar.
The conference was, as Pierre Abélard might have put it, a “sic et non” experience. Lots of good advice – but not a lot of applicable next steps. What makes this all the more urgent is the South African position on medicines reimbursement and intellectual property – which shares many philosophical underpinnings with that other bastion of IP protection – India.
Just as there are issues relative to South Africa’s need for regulatory capacity building, so too is capacity building and investment important for the future of intellectual property issues. One frightening passage in South Africa’s proposal states, “The Draft Policy proposes adopting strict patenting rules to “exclude diagnostic, therapeutic and surgical methods from patentability, including new uses of known products, as is the case under the TRIPS Agreement.” Not a good start for a nation that wants to be the “s” added to BRICS.
Further, South African law currently provides no regulatory data protection and the Draft Policy does not include any. Moreover, it explicitly rejects the utility of “blanket data protection” for innovator data and emphasizes the importance of “access to knowledge.” Uh oh. And, of course, there’s a magic rain dance call for compulsory licensing.
The Draft Policy encourages the use of parallel importation to improve access to medicines. Been there. Done that. A clear and present risk to patients.
And these are only a few examples. It gets worse.
The policy disconnect is profound. In April, South Africa sent a sizable delegation to the mega BIO convention with the message that they were “open for business,” and aspires to be a player in global life sciences.
At the same time, local generics manufacturers and the usual suspect activists are whipping up frenzy over intellectual property issues. The same old song. And a dangerous one considering that Scientific American ranks South Africa in the bottom 20% of its life science/innovation index.
(PS/ Under the current regulatory regime, it takes the MCC between 3-5 years after EMA or FDA approval t bring innovative therapeutics to South African patients – the slowest among “advanced” MEA nations. What’s wrong with this picture?)
Rather than looking to emulate the EMA, perhaps South Africa should adopt a reference basket of maybe five to six countries. A good model is Singapore. Everyone’s favorite city-state reviews seven countries—USA, Canada, Australia, NZ, Japan, Switzerland and the EMA. If any two of the seven have approved, then approval is basically a formality.
Talk is cheap.
There is nothing like returning to a place that remains unchanged to find the ways in which you yourself have altered. – Nelson Mandela
At CMPI’s recent Capital Hill conference ,Personalized Medicine and Responsible Access to Pain Medication, Dr. Charles Inturrisi, professor of pharmacology at the Weill Cornell Medical College, laid down the gauntlet:
I want to make a distinction that really does make a difference. And this is the distinction between efficacy and effectiveness. We know that opioids can provide analgesia for some chronic pain patients. We don’t know what percentage but we know that some, and you’ve heard from them this morning, at least one of them. But we also know the treatment outcomes with opioids are variable and not predictable. And this is the take home message if you have to leave. At present, there are no well-validated means of identifying optimal candidates for effective long-term chronic opioid therapy. That’s the problem. That’s the gap in our knowledge. That’s the gap in our evidence base.
We need to learn who will experience good analgesic effectiveness at stable dosages with limited side effects and low risk of abuse. So the critical question there is are there phenotypic or endo-genotypic characteristics that we can associate with better or worse outcomes that will help us to predict which patients might benefit and so that the cost-benefit ratio will be favorable rather than unfavorable.
Now I’m going to talk about personalized medicine in general and in particular. This refers to this emerging concept approach that uses patient-related factors including the phenotype, that is what information you can observe about the patient and a lot of that information now is contained in the electronic medical record. Also genotypic information that you can gain by collecting a sample and it can be either a sample of blood, or in some cases even a sample of saliva and by going through and looking at snips of DNA and creating biomarkers that select optimum medication and dosage for individual patients. It’s been estimated, on average, that prescription drugs are effective for only about half of those who take them. And for some drugs like anticancer drugs and antidepressants, the so-called non-responder rate is even higher.
Personalized medicine can reduce the non-responder rate because you can focus in on individuals who are highly associated with being respondersand you can eliminate the trial and error inefficiencies that inflate healthcare cost.
An audio recording on Dr. Inturrisi’s full comments can be found here (at the 1:03 mark) and the panel discussion that followed here.
On Friday CMS issued its final decision on beta amyloid imaging. It’s similar to their draft decision in that they ignored the recommendations of the medical community that was calling for full coverage for the Appropriate Use Criteria (AUC) population and imposed Coverage with Evidence Development (CED). CMS will only pay for Alzheimer's imaging tests used in clinical research or to exclude Alzheimer's disease when diagnosing patients in narrow circumstances. There are some slight changes from the initial draft, but from a patient access perspective, we are in the same boat as we were previously, which is non-coverage.
The Alzheimer's Association noted that, in the past, it has taken as long as seven years for CMS to move from a CED designation for new medical technologies to full coverage.
CMS’ move is just the latest example of cost-based thinking trumping patient-centric care
And, as per a recent article in BioCentury, there are significant unintended consequences that will impact the future of personalized medicine.
The cost of demonstrating clinical utility, along with the lack of clear or consistent standards, is killing a business model that had made it possible for small companies to commercialize molecular diagnostics quickly and cheaply. The fate of these laboratory-developed molecular diagnostics companies, and especially the conclusions investors draw about the viability of the space, could shape the future of personalized medicine.
According to research by the Tufts Center for the Study of Drug Development, without clinically useful diagnostics, personalized medicine growth will occur at a relatively slow pace.
And personalized medicine represents the future of healthcare around the world.
Let’s cut to the chase, if we are going to take meaningful strides both in addressing Alzheimer’s Disease specifically and in personalized medicine more broadly, we should not rely on Coverage with Evidence Development (CED) criteria in cases where the FDA’s approval process has expressly evaluated and endorsed the use of a drug or biologic in a specific patient population.
Fact: The evidence on amyloid imaging supports coverage for the population as identified by the Amyloid Imaging Task Force through Appropriate Use Criteria (AUC). A task force, convened by the Alzheimer’s Association and the Society of Nuclear Medicine and Molecular Imaging, recommends coverage in this population based on a comprehensive review of the literature and expert consensus.
Fact: CMS currently covers similar PET technologies to aid in the diagnosis of Alzheimer’s Disease and other forms of cognitive decline. The agency has not previously required evidence of health outcome improvement as a condition of such coverage.
Fact: Using CED alone will deny Medicare beneficiaries adequate and rapid access to this technology, as the path to implementation is unclear. Such uncertainty in the reimbursement process strongly dis-incentivizes future investments in research and development. And without innovation there will not be advances in personalized medicine.
Wither “sustainable innovation?”
Why even bother with expedited review and similar FDA pathways? Clearly closer FDA/CMS coordination is required to address both the will of Congress – and the future of American healthcare.
Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.
